Bone marrow transplantation in patients with thalassemia.
We reviewed the results of transplantation of allogeneic marrow from HLA-identical donors in patients with beta-thalassemia who were less than 16 years old. Among the 222 consecutive patients who had received transplants since 1983, survival and event-free-survival curves leveled off about one year after transplantation, at 82 and 75 percent, respectively. Pretransplantation clinical characteristics were examined for their impact on survival, event-free survival, and the recurrence of thalassemia in the 116 consecutive patients who were treated with our current regimen, in use since June 1985. In a multivariate analysis, portal fibrosis and either the presence of hepatomegaly or a history of inadequate chelation therapy were significantly associated with reduced probabilities of survival and event-free survival. The patients were divided into three classes on the basis of the presence of hepatomegaly or portal fibrosis (class 1 had neither factor, class 2 had one, and class 3 had both). For class 1 patients the three-year probabilities of survival, event-free survival, and recurrence were 94, 94, and 0 percent, respectively. For class 2 patients the probabilities were 80, 77, and 9 percent, and for class 3 patients 61, 53, and 16 percent. We conclude that for patients under 16 years of age, transplantation of bone marrow from an HLA-identical donor offers a high probability of complication-free survival, particularly if they do not have hepatomegaly or portal fibrosis.
Lucarelli
G
G
Division of Hematology, Ospedale di Pesaro, Italy.
Galimberti
M
M
Polchi
P
P
Angelucci
E
E
Baronciani
D
D
Giardini
C
C
Politi
P
P
Durazzi
S M
SM
Muretto
P
P
Albertini
F
F
D000293:Adolescent; D016026:Bone Marrow Transplantation; D002648:Child; D002675:Child, Preschool; D006529:Hepatomegaly / Q000150:complications; D006801:Humans; D007223:Infant; D008103:Liver Cirrhosis / Q000150:complications; D015999:Multivariate Analysis; D012008:Recurrence; D015996:Survival Rate; D013789:Thalassemia / Q000150:complications / Q000401:mortality / Q000601:surgery*